摘要

The chance to selectively intervene and stop the development of any gene-dependent disease in different organs and pathologies makes siRNA a potential therapeutic agent. However, serious issues remain to be addressed before the real therapeutic use of siRNA. The poor pharmacokinetic properties of siRNA, its short half-life, its low in vivo stability, its fast elimination by renal excretion and its low transfection efficiency complicate the use of siRNA as a therapeutic molecule. In this review, we will describe the latest and most advanced approaches and strategies undertaken to address these limitations and improve siRNA delivery and its gene-silencing efficacy as well as the prospects for its therapeutic application.